Accelerating Cell & Gene Therapy Product Development via NGS
November 15, 2023
Urszula Cendrowska
An increasing number of novel biotherapeutics, such as cell and gene therapies (CGT), are being developed to treat patients with genetic disorders. For these treatments, speed and safety are critical for success. The biopharma industry is witnessing a significant transformation with the advent of Next-generation sequencing (NGS), which efficiently overcomes the limitations of traditional approaches in product development and characterization. Genedata Selector® is an end-to-end platform that enables biopharmaceutical organizations to perform various NGS-based assays in-house. Our commitment to facilitating knowledge exchange and expert community building in the biopharma industry is reflected in our organization of regular industry workshops. These meetings bring together NGS experts to discuss this fast-evolving area and the important role NGS plays in advancing drug development and manufacturing.
On November 9th, 2023, Genedata Selector hosted its 8th Industry Workshop. The virtual event focused on the implementation of NGS in the development and manufacturing of novel biopharmaceuticals, including cell therapies. The workshop brought together various scientific leaders to exchange knowledge and insights on best practices, creating an environment for mutual learning. The 8th edition of the Open Forum was co-hosted by the Astellas Institute for Regenerative Medicine (AIRM), which recently licensed Genedata Selector to support in confirming the biosafety of cell therapies using NGS-based assays within a validated environment. The event featured presentations from industry leaders at Astellas, Umoja Biopharma, Merck MSD, Bristol Myers Squibb, Takeda, and Genedata. The talks centered around various topics such as the significance of utilizing NGS for the biosafety testing of biopharmaceuticals and NGS as a promising Multi-Attribute Method (MAM) for assessing critical quality attributes (CQAs) of Advanced Therapy Medicinal Products (ATMP). The event was attended by participants from 30 companies, and it provided a highly interactive platform for attendees to discuss and share their expertise. Here are some key takeaways from this successful workshop.
The meeting began with a warm welcome and introduction from Marco de Cecco of Astellas AIRM. He explained AIRM’s motivation behind implementing NGS for characterizing and releasing cell therapy products and overcoming the limitations of traditional biosafety testing. He emphasized the significance of utilizing NGS for Adventitious Agent Detection (AAD) as a CQA to safeguard all GMP-manufactured cell products. Marco concluded his introduction by highlighting the virtuous collaboration with Genedata Selector and the platform's ability to integrate, analyze, and share project-related data in a regulated environment for improved decision-making and faster time to market.
The presentation delivered by our Special Guest Richard Rogers, Director of Process Analytics at Umoja Biopharma, focused on the development and evolution of the Multi-Attribute-Method (MAM) approach. The MAM approach was originally developed from a Mass Spectrometry (MS) project in Quality Control (QC) at Amgen. Richard explained that the primary objective of implementing the MAM approach in product characterization is to replace traditional QC release assays. He demonstrated how MAM can replace many traditional release methods, including immunoassays, ELISA, and HPLC. He also emphasized its current use in large-molecule and cell-based therapies. Richard concluded his presentation with a forward-looking perspective, underscoring the promise and potential of utilizing NGS as a MAM to characterize and release various biotherapeutics, particularly for cell and gene therapy.
The Open Forum provided a glimpse into the advanced capabilities of the Genedata Selector platform for quality control in cell therapy production. Anukana Bhattacharjee, Scientific Consultant at Genedata, presented an interactive demo showcasing how NGS can be used as a multi-attribute method (MAM) for assessing critical quality attributes (CQAs) during cell therapy production and manufacturing. Using data obtained from the Fraunhofer Institute for Interfacial Engineering Biosafety and Biotechnology IGB in Stuttgart, Germany, Anukana illustrated how Genedata Selector supports NGS-based adventitious agent detection, correct variant introduction, and differential gene expression assessment. The audience had the opportunity to view the outcome of this analysis as a single, unified overview of QC results across all run NGS-based analyses. Using customized and standardized Playbooks, Anukana demonstrated how to turn complex NGS assays into automated and streamlined workflows. Additionally, she showed how Genedata Selector provides actionable go/no-go signals for better-informed decision-making regarding critical quality testing of cell therapy products.
Finally, Vanessa Sarathy, Associate Principal Scientist from MSD Research Laboratories, delivered a presentation on the importance of using Next-Generation Sequencing (NGS) as a biosafety testing tool for detecting viruses in biopharmaceuticals. She explained the several steps involved in Viral Metagenomic Analysis VMA, from sample processing to bioinformatics analysis. In the second part of her talk, she presented a case study comparing different bioinformatic tools for NGS-based biosafety analysis. The tools were tested for their total hits, breadth of genome coverage, and detection of spiked-in viruses. Results showed that the tested tools had similar limits of detection and performance. At the end of her talk, Vanessa highlighted that further automation of several types of NGS-based analyses is required to facilitate the GMP assays.
After each presentation, a lively Q&A session took place where Open Forum attendees were able to submit their questions or dial in and share their perspectives with the speakers and the other participants.
The event concluded with a panel discussion featuring our speakers and two additional guest panelists – Athea Vichas, Senior Scientist at Bristol Myers Squibb, and Yingying Zhang, Director of Analytical Development at Takeda. During the discussion, the panelists shared their perspectives on the applications of NGS in drug development. During the discussion, the panel and the audience agreed that variant detection through NGS-based assays is often considered by organizations seeking to streamline their drug development and manufacturing. They also highlighted the key benefits of integrating enterprise IT with NGS testing. Finally, they briefly discussed what would assist the QA teams in initiating NGS validation and GMP implementation. The panel discussion was highly interactive with numerous live polls that allowed all the attendees to participate and shape the discussion. The polls provided interesting and valuable insights based on the responses of participating voters. In addition to the polls, participants could dial in, ask specific questions, and engage in the discussion with our panelists.
Our Genedata Selector Industry Workshops aim to bring together scientific experts from leading biopharmaceutical companies to exchange knowledge regarding NGS-based assays for biopharmaceutical R&D. These thematic events have gained increasing popularity among the ever-growing community, providing participants with the opportunity to learn and share their perspectives on best practices for utilizing NGS in biosafety assessment, advanced biopharmaceuticals development, and cell line development. Join our exclusive community and register your interest today to ensure you do not miss any future meetings.
